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Combination of Ibuprofen, G-CSF and Plerixafor as Stem Cells Mobilization Regimen in Patients Affected by X-CGD (XCGD-MOBI)

Primary Purpose

Chronic Granulomatous Disease X-linked (X-CGD)

Status

Recruiting

Phase

Phase 2

Locations

Italy

Study Type

Interventional

Intervention

Ibuprofen

Myelostim

Mozobil

About this trial

This is an interventional basic science trial for Chronic Granulomatous Disease X-linked (X-CGD) focused on measuring Chronic granulomatous disease X-linked, Ibuprofen, Mobilization regimen, Gene Therapy

Eligibility Criteria

18 Years - 45 Years (Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

Genetic diagnosis of X-CGD
18-45 years of age
Karnofsky Index > 80 %
Adequate cardiac, renal, hepatic and pulmonary function.
Negative thrombophilic screen and negative history for previous thrombotic events
Written informed consent

Exclusion Criteria:

Previous Bone Marrow Transplantation or previous Gene Therapy.
Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long-acting agents).
Ongoing IFN-γ treatment (within 4 weeks).
Symptomatic inflammatory bowel disease.
Symptomatic viral, bacterial, or fungal infection within 6 weeks of eligibility
Neoplasia (except local skin cancer) or history of "familial" cancer
Myelodysplasia or other serious hematological disorder
History of uncontrolled seizures and deep venous thrombosis
Other systemic disease judged as incompatible with the procedure
Positivity for HIV and/or HCV RNA and/or HbsAg and/or HBV DNA
Active alcohol or substance abuse within 6 months of the study.
Contraindications to IBU, G-CSF, Plerixafor or Pantoprazole administration

Sites / Locations

Ospedale Pediatrico Bambino GesùRecruiting
Ospedale San RaffaeleRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

XCGD mobilization

Arm Description

Treatment with combination of Ibuprofen, Myelostim and Mozobil

Outcomes

Primary Outcome Measures

Percentage of patients experiencing adverse events

Percentage of patients experiencing adverse events, as assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse events (CTCAe v3.0, 2006) (all grades).

Number of CD34+ collected per body weight after the last LP

Cytofluorimetric analysis for CD34 on PB and on collected PBSC to calculate the number of CD34+ cells collected per kg body weight. The analysis will be performed at the end of the LP(s) (Day 21-24)

Secondary Outcome Measures

Change in number of CD34+ cells in PB before and after administration of Ibuprofen

Cytofluorimetric analysis to determine the number of CD34+ cells present in PB on day 6 and 7 compared to before the administration of Ibuprofen

Transduction efficiency

Efficient transduction of mobilized HSPC with a lentiviral vector encoding for a corrective cDNA of the human gp91phox gene. Frequency and Vector Copy Number tested by PCR.

DHR (dihydrorhodamine) test in myeloid progeny

Correction of the functional defects in the differentiated myeloid progeny

Functional characterization of mobilized CD34+ cells.

Phenotype analysis (FACS).

Functional characterization of mobilized CD34+ cells.

Clonogenic activity (CFU-C) before and after transduction.

Functional characterization of mobilized CD34+ cells.

Repopulating activity of mobilized CD34+ cells in immunodeficient mice.

Full Information

NCT ID

NCT03055247

First Posted

July 21, 2016

Last Updated

October 27, 2022

Sponsor

IRCCS San Raffaele

Collaborators

Fondazione Telethon

1. Study Identification

Unique Protocol Identification Number

NCT03055247

Brief Title

Combination of Ibuprofen, G-CSF and Plerixafor as Stem Cells Mobilization Regimen in Patients Affected by X-CGD

Acronym

XCGD-MOBI

Official Title

A Multicentric, Exploratory, Non-randomised, Non-controlled, Prospective, Open-label Phase II Study Evaluating Safety and Efficacy of IBU, G-CSF and Plerixafor as Stem Cell Mobilization Regimen in Patients Affected by X-CGD

Study Type

Interventional

2. Study Status

Record Verification Date

October 2022

Overall Recruitment Status

Recruiting

Study Start Date

November 6, 2015 (Actual)

Primary Completion Date

July 13, 2023 (Anticipated)

Study Completion Date

July 18, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

IRCCS San Raffaele

Collaborators

Fondazione Telethon

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

This is a phase II exploratory study conducted to evaluate the safety and efficacy of the combination of Ibuprofen, G-CSF and Plerixafor as stem cell mobilization regimen in patients affected by X-CGD.

Detailed Description

We designed a mobilization trial with the aim of collecting a sufficient number of HSPC in X-CGD patients; it is well known that this procedure is challenging for these patients, potentially due to functional defects induced by their chronic inflammatory state. The combination of G-CSF and Plerixafor is considered state of the art for HSPC harvest in gene therapy trials; we considered to add a non-steroidal inflammatory drug to increase HSPC mobilization and reduce inflammation that could have a role in altering HSPC content. If this trial confirms the synergistic effect of the three drugs under investigation, such a regimen will be considered for a HSPC mobilization in future gene therapy trial for X-CGD patients.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Chronic Granulomatous Disease X-linked (X-CGD)

Keywords

Chronic granulomatous disease X-linked, Ibuprofen, Mobilization regimen, Gene Therapy

7. Study Design

Primary Purpose

Basic Science

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

3 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

XCGD mobilization

Arm Type

Experimental

Arm Description

Treatment with combination of Ibuprofen, Myelostim and Mozobil

Intervention Type

Drug

Intervention Name(s)

Ibuprofen

Intervention Description

Ibuprofen: 3 mg/kg tid (total daily dose: 9 mg/kg); administered orally from day 1 to day 5 and then from day 14 to the day before the last LP.

Intervention Type

Drug

Intervention Name(s)

Myelostim

Intervention Description

Myelostim (G-CSF): 5 µg/kg bid (total daily dose 10 µg/kg); administered subcutaneously from day 19 to the day of the last LP.

Intervention Type

Drug

Intervention Name(s)

Mozobil

Intervention Description

Mozobil (Plerixafor): 0,24 mg/kg daily. When CD34+ are ≥ 10 /μL Plerixafor will be administered subcutaneously from the next day (or from day 24 if CD34+ are < 10 /μL) to the day of the last LP.

Primary Outcome Measure Information:

Title

Percentage of patients experiencing adverse events

Description

Percentage of patients experiencing adverse events, as assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse events (CTCAe v3.0, 2006) (all grades).

Time Frame

up to 30 days after the last LP

Title

Number of CD34+ collected per body weight after the last LP

Description

Cytofluorimetric analysis for CD34 on PB and on collected PBSC to calculate the number of CD34+ cells collected per kg body weight. The analysis will be performed at the end of the LP(s) (Day 21-24)

Time Frame

Day 21-24

Secondary Outcome Measure Information:

Title

Change in number of CD34+ cells in PB before and after administration of Ibuprofen

Description

Cytofluorimetric analysis to determine the number of CD34+ cells present in PB on day 6 and 7 compared to before the administration of Ibuprofen

Time Frame

Day 6 and day 7

Title

Transduction efficiency

Description

Efficient transduction of mobilized HSPC with a lentiviral vector encoding for a corrective cDNA of the human gp91phox gene. Frequency and Vector Copy Number tested by PCR.

Time Frame

Through study completion, an average of 1 year

Title

DHR (dihydrorhodamine) test in myeloid progeny

Description

Correction of the functional defects in the differentiated myeloid progeny

Time Frame

Through study completion, an average of 1 year

Title

Functional characterization of mobilized CD34+ cells.

Description

Phenotype analysis (FACS).

Time Frame

Through study completion, an average of 1 year

Title

Functional characterization of mobilized CD34+ cells.

Description

Clonogenic activity (CFU-C) before and after transduction.

Time Frame

Through study completion, an average of 1 year

Title

Functional characterization of mobilized CD34+ cells.

Description

Repopulating activity of mobilized CD34+ cells in immunodeficient mice.

Time Frame

Through study completion, an average of 1 year

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

45 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Genetic diagnosis of X-CGD 18-45 years of age Karnofsky Index > 80 % Adequate cardiac, renal, hepatic and pulmonary function. Negative thrombophilic screen and negative history for previous thrombotic events Written informed consent Exclusion Criteria: Previous Bone Marrow Transplantation or previous Gene Therapy. Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long-acting agents). Ongoing IFN-γ treatment (within 4 weeks). Symptomatic inflammatory bowel disease. Symptomatic viral, bacterial, or fungal infection within 6 weeks of eligibility Neoplasia (except local skin cancer) or history of "familial" cancer Myelodysplasia or other serious hematological disorder History of uncontrolled seizures and deep venous thrombosis Other systemic disease judged as incompatible with the procedure Positivity for HIV and/or HCV RNA and/or HbsAg and/or HBV DNA Active alcohol or substance abuse within 6 months of the study. Contraindications to IBU, G-CSF, Plerixafor or Pantoprazole administration

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Fabio Ciceri, MD, PhD

Phone

39 02.2643.3903

ciceri.fabio@hsr.it

First Name & Middle Initial & Last Name or Official Title & Degree

Alessandro Aiuti, MD, PhD

Phone

+390226434875

aiuti.alessandro@hsr.it

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Fabio Ciceri, MD, PhD

Organizational Affiliation

Ospedale San Raffaele

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Franco Locatelli, MD, PhD

Organizational Affiliation

Ospedale Pediatrico Bambino Gesù

Official's Role

Principal Investigator

Facility Information:

Facility Name

Ospedale Pediatrico Bambino Gesù

City

Rome

State/Province

Lazio

ZIP/Postal Code

00165

Country

Italy

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Franco Locatelli, MD, PhD

First Name & Middle Initial & Last Name & Degree

Andrea Finocchi, MD

Facility Name

Ospedale San Raffaele

City

Milan

State/Province

Lombardia

ZIP/Postal Code

20132

Country

Italy

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Fabio Ciceri, MD, PhD

First Name & Middle Initial & Last Name & Degree

Alessandro Aiuti, MD, PhD

First Name & Middle Initial & Last Name & Degree

Bernhard Gentner, MD, PhD

First Name & Middle Initial & Last Name & Degree

Maddalena Migliavacca, MD

First Name & Middle Initial & Last Name & Degree

Laura Bellio, MD

12. IPD Sharing Statement

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Combination of Ibuprofen, G-CSF and Plerixafor as Stem Cells Mobilization Regimen in Patients Affected by X-CGD

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